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P183 Burden of illness in school-aged patients with cystic fibrosis (CF) in the united states
  1. J Rubin1,
  2. M Bonafede2,
  3. S Sikirica1,
  4. B Limone2,
  5. N Adolph2,
  6. M Konstan3
  1. 1Vertex Pharmaceuticals Incorporated, Boston, MA, USA
  2. 2Truven Health Analytics, Cambridge, MA, USA
  3. 3Case Western Reserve University School of Medicine and Rainbow Babies and Children’s Hospital, Cleveland, OH, USA


Background and objectives The CF disease burden among school-aged children is not well understood. We sought to describe this burden in patients with CF aged 6–17 years in the United States by comparing their healthcare resource utilisation (HCRU) to that of demographically similar controls without CF.

Methods This retrospective study used administrative claims from the Truven MarketScan Medicaid Multi-State (CAID) and Commercial (COMM) databases. Patients with CF aged 6–17 years were identified as having ≥1 inpatient (IP) or ≥2 outpatient (OP) medical claims ≥30 days apart with primary diagnosis of CF (ICD-9-CM: 277.0x) between 2010 and 2014. Other inclusion criteria were ≥12 months of continuous medical and pharmacy coverage and ≥1 CF-related healthcare encounter during the most recent year of data. Patients were matched 1:3 to non-CF controls by age, gender, race (CAID cohort), geographic region (COMM cohort), insurance plan type and enrollment (calendar year). IP admissions, OP visits and medication use from the most recent year of data (2010–2014) were compared between patients and controls, overall and by age group (6–11 and 12–17 years), using bivariate statistics; chi-square tests were used for categorical variables and t tests and ANOVA for continuous variables.

Results The CAID cohort included 1264 patients with CF and the COMM cohort 2400; all were matched 1:3 to controls (mean [SD] age of patients and controls: CAID, 11.4 [3.5]; COMM, 11.9 [3.5]). Annual hospitalisation rates were 22-fold (CAID) to 32-fold (COMM) higher in the CF cohorts, with lengths of stay nearly twice that of matched controls (Table). Annual OP visit rates were 3.1-fold (CAID) and 3.5-fold (COMM) higher in the CF cohorts, and patients filled 5 times as many unique medications and 10 times as many total prescriptions per year as controls. While patients with CF aged 12–17 years generally had higher HCRU than those aged 6–11, trends and magnitude difference vs controls within each age group were similar.

Conclusion HCRU was higher in patients with CF aged 6–17 years than in demographically similar children without CF, illustrating significant disease burden and a need for better treatment options for this population.

Sponsored by Vertex Pharmaceuticals Incorporated

Please refer to page A272 for declarations of interest in relation to abstract P183.

Abstract P183 Table 1

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